Guselkumab for Pediatric Ulcerative Colitis
(QUASAR Jr Trial)
Recruiting in Palo Alto (17 mi)
+67 other locations
Age: < 18
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Janssen Research & Development, LLC
Disqualifiers: Severe colitis, Crohn's disease, others
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Approved in 2 Jurisdictions
Trial Summary
What is the purpose of this trial?This trial is testing guselkumab, a medication that reduces inflammation, in children with severe ulcerative colitis who have already shown improvement with earlier treatment. It works by blocking a protein to reduce immune system-related swelling and irritation. Guselkumab has shown benefit in treating inflammatory conditions, with reports of substantial improvement in patients.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What data supports the effectiveness of the drug Guselkumab for pediatric ulcerative colitis?
Guselkumab has been studied for its effectiveness in adults with ulcerative colitis, showing promise in patients who did not respond well to other treatments. While specific data for children is limited, similar biologic drugs have been used successfully in pediatric ulcerative colitis, suggesting potential benefits.
12345How is the drug Guselkumab different from other treatments for pediatric ulcerative colitis?
Guselkumab is unique because it targets a specific protein involved in inflammation, which is different from other treatments like steroids or anti-TNF drugs that have broader effects. This targeted approach may offer a new option for children with ulcerative colitis who do not respond to existing therapies.
34567Eligibility Criteria
This trial is for children and teenagers with moderately to severely active ulcerative colitis, a type of inflammatory bowel disease. Participants must have shown some response to earlier treatment phases to qualify.Inclusion Criteria
I have a confirmed diagnosis of ulcerative colitis with biopsy proof.
My ulcerative colitis is moderate to severe, confirmed by specific tests.
My health is stable according to my recent medical check-up.
+2 more
Exclusion Criteria
My ulcerative colitis affects only my rectum or less than 20cm of my colon.
I have a stoma.
I have not had bowel surgery in the last 6 months or any abdominal surgery in the last 3 months.
+1 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Open-label Induction
Participants receive guselkumab intravenously or subcutaneously based on their body weight during the 12-week open-label induction phase
12 weeks
Double-blind Maintenance
Week 12 responders are randomized to receive guselkumab dose regimen 1 or 2 subcutaneously based on their body weight up to Week 56
44 weeks
Open-label Maintenance
Week 12 non-responders enter an open-label maintenance phase to receive guselkumab subcutaneously based on their body weight up to Week 56
44 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
12 weeks
Participant Groups
The study tests the effectiveness of Guselkumab, a medication, in young patients with ulcerative colitis. It compares Guselkumab against a placebo (a substance with no therapeutic effect) over time.
5Treatment groups
Experimental Treatment
Group I: Open-label Maintenance Phase: Guselkumab SCExperimental Treatment1 Intervention
Week 12 non-responders will not be randomized and will enter an open-label maintenance phase to receive guselkumab SC dosing regimen based on their body weight up to Week 56.
Group II: Open-label Induction Phase: Guselkumab Subcutaneously (SC)Experimental Treatment1 Intervention
Participants will receive a guselkumab dose SC based on their BW during the 12-week open-label induction phase.
Group III: Open-label Induction Phase: Guselkumab Intravenously (IV)Experimental Treatment1 Intervention
Participants will receive a guselkumab dose IV based on their body weight (BW) during the 12-week open-label induction phase.
Group IV: Double-blind Maintenance Phase: Guselkumab Dose Regimen 2Experimental Treatment1 Intervention
At the end of the induction phase, Week 12 responders will be randomized into the double-blind maintenance phase to receive guselkumab dose regimen 2 SC based on their BW up to Week 56.
Group V: Double-blind Maintenance Phase: Guselkumab Dose Regimen 1Experimental Treatment1 Intervention
At the end of the induction phase, Week 12 responders will be randomized into the double-blind maintenance phase to receive guselkumab dose regimen 1 SC based on their BW up to Week 56.
Guselkumab is already approved in United States, European Union for the following indications:
🇺🇸 Approved in United States as Tremfya for:
- Moderate to severe plaque psoriasis
- Psoriatic arthritis
- Moderately to severely active ulcerative colitis
🇪🇺 Approved in European Union as Tremfya for:
- Moderate to severe plaque psoriasis
- Psoriatic arthritis
- Moderately to severely active ulcerative colitis
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
NYU Langone Long Island Clinical Research AssociatesLake Success, NY
NYU Langone HealthLake Success, NY
Riley Hospital for ChildrenIndianapolis, IN
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Who Is Running the Clinical Trial?
Janssen Research & Development, LLCLead Sponsor
References
Guselkumab plus golimumab combination therapy versus guselkumab or golimumab monotherapy in patients with ulcerative colitis (VEGA): a randomised, double-blind, controlled, phase 2, proof-of-concept trial. [2023]Despite the introduction of new monoclonal antibodies and oral therapies for the treatment of ulcerative colitis, clinical remission rates remain low, underscoring the need for innovative treatment approaches. We assessed whether guselkumab plus golimumab combination therapy was more effective for ulcerative colitis than either monotherapy.
Guselkumab in Patients With Moderately to Severely Active Ulcerative Colitis: QUASAR Phase 2b Induction Study. [2023]The QUASAR Phase 2b Induction Study evaluated the efficacy and safety of guselkumab, an interleukin-23p19 subunit antagonist, in patients with moderately to severely active ulcerative colitis (UC) with prior inadequate response and/or intolerance to corticosteroids, immunosuppressants, and/or advanced therapy.
New Therapeutic Strategies Are Associated With a Significant Decrease in Colectomy Rate in Pediatric Ulcerative Colitis. [2023]We evaluated the impact of immunosuppressants (IS) and antitumor necrosis factor (TNF) introduction on long-term outcomes of ulcerative colitis (UC) in a large population-based pediatric-onset cohort.
Induction and maintenance therapy with infliximab for children with moderate to severe ulcerative colitis. [2022]We evaluated the efficacy and safety of infliximab for inducing and maintaining benefit in children with moderately to severely active ulcerative colitis (UC).
Special considerations for biologic medications in pediatric ulcerative colitis. [2020]Introduction: More extensive disease, high rates of corticosteroid refractory and dependent disease, and the potential impact of disease on growth and development differentiate inflammatory bowel disease in children from adults. This is particularly evident in ulcerative colitis where pancolitis predominates, success of mesalamine alone in achieving remission is less than 50%, and there is a high need for immunomodulator or biologic therapies.Areas Covered: This review describes the use of infliximab, adalimumab, golimumab, and vedolizumab in the treatment of children with ulcerative colitis but is limited in scope due to the paucity of controlled clinical trials. A search of existing literature with keywords of these specific biological therapies as well as 'pediatric', 'ulcerative colitis,' and 'inflammatory bowel disease' was used to complete this review.Expert Opinion: Therapeutic drug monitoring has become standard of care when assessing dosing and changes in therapy and will play a role in future treatment planning.
Tofacitinib for a Child with Refractory Steroid-Dependent Ulcerative Colitis: A Case Report and Review of the Literature. [2021]BACKGROUND Ulcerative colitis (UC) is a chronic autoimmune inflammatory disease of the colon that infrequently affects children. The disease requires immunosuppressive therapy to achieve remission and keep the disease in remission. Currently, many therapies are approved for use in pediatric patients with UC, including steroid, 5-aminosalicylic acid (5-ASA), azathioprine, and biologic therapy with anti-tumor necrosis factor (TNF) inhibitors. Despite their efficacy, many patients have refractory severe disease that fails therapy and may require surgical interventions. Recently, the small molecule Janus Kinase (JAK) inhibitor tofacitinib has been approved for moderate to severe UC that fails biologic therapy in adults. However, the safety and efficacy of this drug has not been tested in pediatric UC patients. CASE REPORT We describe a case of a 13-year-old girl with 2-year history of severe UC who had secondary loss response to both infliximab and adalimumab over 2 years, despite adequate trough serum drug levels and the concomitant use of azathioprine. She was also dependent on steroid to control her disease. Infectious work-ups were always negative for infectious organisms. She was then successfully treated with tofacitinib 5 mg orally twice daily. She went into complete clinical, endoscopic, and steroid-free remission. CONCLUSIONS This case report highlights the safety and efficacy of tofacitinib in pediatric patients with severe refractory UC, potentially avoiding proctocolectomy in this young patient population. Future research should study the role of tofacitinib in patients with moderate to severe UC in children.
Subcutaneous Golimumab in Pediatric Ulcerative Colitis: Pharmacokinetics and Clinical Benefit. [2018]Current treatments for pediatric ulcerative colitis (UC) are limited. We evaluated the pharmacokinetics and clinical benefits of subcutaneous golimumab, an anti-tumor necrosis factor agent, in moderately-to-severely active pediatric patients with UC refractory to conventional therapy.